Clinical trial
A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene (GLOBE1 βAS3 Modified Autologous CD34+ Cells) in Patients With Sickle Cell Disease (SCD)
NCT03964792 is registered in ClinicalTrials.gov with Assistance Publique - Hôpitaux de Paris as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene (GLOBE1 βAS3 Modified Autologous CD34+ Cells) in Patients With Sickle Cell Disease (SCD)
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Phase
- Phase 1/2
- Overall status
- Completed
- Start date
- November 12, 2019
- Completion date
- January 23, 2024
- Enrollment
- 6 (actual)
- Sites
- 1
Conditions
As the registry lists them; a condition with its own page is linked.
sickle cell disease